Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.

Publikation

Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale.

Wissenschaftlicher Artikel/Review - 28.08.2024

van Kooten Harmke A, Horton Mike C, Wenninger Stephan, Babačić Haris, Schoser Benedikt, Lefeuvre Claire, Taouagh Najib, Laforet Pascal, Segovia Sonia, Diaz-Manera Jordi, Claeys Kristl G, Mongini Tiziana, Musumeci Olimpia, Toscano Antonio, Hundsberger Thomas, Brusse Esther, van Doorn Pieter A, van der Ploeg Ans T, van der Beek Nadine A M E, European Pompe Consortium study group on outcome measures

Zitation

van Kooten H, Horton M, Wenninger S, Babačić H, Schoser B, Lefeuvre C, Taouagh N, Laforet P, Segovia S, Diaz-Manera J, Claeys K, Mongini T, Musumeci O, Toscano A, Hundsberger T, Brusse E, van Doorn P, van der Ploeg A, van der Beek N, European Pompe Consortium study group on outcome measures. Improving outcome measures in late onset Pompe disease: Modified Rasch-Built Pompe-Specific Activity scale. Eur J Neurol 2024; 31:e16397.

Art

Wissenschaftlicher Artikel/Review (Englisch)

Zeitschrift

Eur J Neurol 2024; 31

Veröffentlichungsdatum

28.08.2024

eISSN (Online)

1468-1331

Seiten

e16397

Kurzbeschreibung/Zielsetzung

The Rasch-Built Pompe-Specific Activity (R-PAct) scale is a patient-reported outcome measure specifically designed to quantify the effects of Pompe disease on daily life activities, developed for use in Dutch- and English-speaking countries. This study aimed to validate the R-PAct for use in other countries.